Frequently Asked Questions
What are promising therapies?
These are any treatment ideas that have some scientific validation but are not yet part of mainstream medicine. Thus your physician is unlikely to tell you about them. For example, for cancer the treatment might be a substance that can kill cancer but not normal cells in the test tube. It might be able to cure cancer in animals bearing tumors. It may even have been tried in humans with some success and available as anecdotal reports on the web or in a peer reviewed journal. Or there may be a single arm clinical study published or even a small randomized trial with encouraging results. There may be epidemiological data showing that patients who happened to have received this treatment have a lower cancer incidence, or their outcomes are better if they were to get cancer even when treated with standard therapy. In most cases, there will be no randomized placebo controlled large-scale (phase III trial) data since, if this were to exist and be positive, the treatment would be part of the standard of care and thus no longer be classified as simply promising.
Where does GlobalCures find ideas for promising therapies?
A database, initially focused on cancer, has been started over the last several years, with inputs from many sources: Google, the media, Pubmed, patients and physicians. We have probed in some detail several hundred ideas and communicated with dozens of individuals. An expanding circle of scientific advisors, in cancer research and outside of cancer, provide the input on these ideas. The resulting database is continuously growing and evolving, benefitting all three GlobalCures programs (FIND, SHARE and FUND).
How does GlobalCures prioritize treatments to develop and to place in its FIND, SHARE and FUND programs?
As noted, the singular focus for GlobalCures trials will be financial orphans. In addition, the following criteria will be used for prioritizing promising therapies from the database it has created:
Is there scientific merit?
- How strong is the science underlying the treatment? Is the mechanism of action of the treatment understood?
- As explained elsewhere, GlobalCures believes that an immunotherapy framework will form the basis for a cancer cure. Thus ideas that fit into such a framework will receive top billing.
Is there human data?
- Human data (especially efficacy data) always trumps animal data which in turn trumps in vitro data. Moreover, phase II data is better than phase I data.
Could trials be implemented immediately?
- This is critical. If the drug or treatment is not available or will require a lengthy approval process, it is prioritized lower.
What are the chances the treatment will lead to a substantial advance versus an incremental improvement in outcomes or in quality of life?
- GlobalCures aims to make big strides in treatment outcomes. Ideas falling into this mindset will be prioritized higher. Overall cost of the therapy (once the above conditions are satisfied)
- The cheaper, the better.
In keeping with its core mission, GlobalCures will not assess potential for profitability of the therapy. This factor will simply not enter into the equation.
Is GlobalCures biased towards alternative or unconventional therapies?
Not at all, but it will take them seriously. GlobalCures' research will either validate or disprove some of the more promising therapies that fall under this category. A negative result will also have considerable value, saving time and money, especially to patients with cancer, who can ill afford to try therapies not known to be efficacious. It should be noted that some of GlobalCures' candidates are as "targeted" as the newest cancer therapies, but they have not been tested rigorously in humans.
Why is this an ideal time to embark on such an approach?
A number of reasons make this an opportune time to move the GlobalCures mission forward:
The “omics” revolutions: we now know what to measure i.e. the parts list in genomics, gene profiling, proteomics, metabolomics, glycomics, etc is becoming available. These advances are allowing the medical community to start individualizing therapy. It is a perfect time to incorporate these methods into the drug development process itself, thus saving cost, time and avoiding needless toxicity. GlobalCures will do so in the studies it designs and sponsors.
New technologies offering cheap and accurate imaging as well as ways of measuring blood and physiological biomarkers, including an increasing number of self-monitoring gadgets are rapidly appearing on the market.
The existence of a rich array of mechanism-based generic drugs with more coming off patent every day continues to enlarge the list of financial orphans.
The Internet and its search capabilities, as well as the advent of social networking, offer a powerful vehicle for generating and implementing ideas (e.g. SHARE) i.e. crowdsourcing at its best.
A rapidly growing population of engaged, well-informed and vocal patients and patient advocates “directing” the course of research, via fund-raising around disease specific, translational goals or through sharing of medical data is available to tap into for the first time in human history through social media services.
Why is there a need to conduct rigorous trials on promising therapies?
Without such trials, one may never know if the drugs/therapies in question are really working or not. They may work for a while and then fail later. They may work best in conjunction with standard chemotherapy. They may have unexpected side-effects. They may work only in a sub-group of cancer patients. These are the types of questions that will be answered by the clinical trials that GlobalCures will conduct. Most importantly, physicians will be reluctant to prescribe these therapies without such data, i.e. potential patient benefit versus risk of the therapy. In addition, physicians worry about losing their license or being sued for prescribing unvalidated therapies; they may get derided by their colleagues, and they may not understand the rationale for the therapy or its interaction with more conventional therapies in the absence of rigorous data.
Will GlobalCures' efforts duplicate those of drug companies?
No. GlobalCures' efforts will be complementary to those in biotech and pharmaceutical companies. Each will play a critical role in the broad need for drug development.
How do GlobalCures and pharmaceutical companies differ in their approach to developing new treatments?
Here is how a drug is developed by the pharmaceutical industry, which spends over $65 billion annually for research and development of new drugs. First, laboratory research identifies substances that cure or ameliorate disease in animals or in the “test-tube”. These promising “leads” are then subjected to rigorous testing in human clinical trials, first to test for toxicity and then for efficacy. If a substance shows that it can treat disease in humans better than what is currently available and the side-effects are tolerable, the substance (now usually referred to as a drug) may be approved by the Food and Drug Administration (FDA) for human use.
The process is inefficient, time consuming, and expensive. Therefore pharmaceutical companies, as for-profit entities, must recoup their investment and so they make a formal return on investment (ROI) calculation prior to embarking on such an enterprise.
A number of factors enter into this calculation. The intellectual property on the treatment/drug being developed must be rock solid, so that they have price exclusivity for a number of years. The market size must be sufficiently large and the population to which the drug is being marketed must have the ability to pay.
The chance that the drug will succeed in clinical studies has be assessed and this will depend on a range of factors, such as how well is the disease pathophysiology understood, how specific is the target being hit for that disease, and so. If all checks out, the drug is developed. If the ROI is judged to be inadequate, the treatment becomes a “financial orphan”. A more detailed explanation is offered in here.
That is where GlobalCures comes in. It looks for these “financial orphans” and adopts them! It also creates combinations of such ideas that might improve efficacy or reduce toxicity. It believes that financial orphans exist for a large number of unmet medical needs, including cancer. Examples include generic, FDA approved drugs, which are often quite inexpensive, in use for non-cancer indications. Other categories of financial orphans include dietary supplements and herbal products available over the counter, or lifestyle manipulations, such as diet, stress control or exercise. Note that the application of these methods is for all diseases, common and rare ones. It is sometimes perceived that GlobalCures is oriented towards the latter; it is not.
Can you give examples of how GlobalCures might partner with for-profit drug companies?
A GlobalCures drug candidate may be added onto a currently accepted regimen. In this case, a partnership with the pharmaceutical company manufacturing the currently used drug will be considered. A fledgling biotech company may not have the funds to build biomarkers into its Phase I trial that would make the trial more informative – i.e. provide what is referred to in the industry as a "proof-of-concept" study. Is the intended target in the tumor tissue being affected by the therapy? GlobalCures might partner with such a company to fund such studies. Another example: there are not enough patients being enrolled in a trial to provide statistical power to evaluate a particular endpoint. GlobalCures might provide resources to fill that need. In exchange for providing such resources, GlobalCures will expect to derive some revenue from the drug company if the drug is approved.
Why donate to GlobalCures?
Reflective of its patient-focused mission, GlobalCures will fund clinical research, whether in a hospital or outpatient setting. By definition it will be focused on patients and thus have immediate impact. Donations to other organizations often support service programs or university wet-lab research that does not involve patients. In addition, the basic science research supported by many organizations is in aggregate a relatively small amount - less than 10% - of similar research being supported by the National Institutes of Health. Moreover, though such research lays the basis for important future advances, the results will take years to impact patient care. By contrast, an investment in GlobalCures has the potential to improve patients’ lives in the short term. GlobalCures prioritizes clinical studies that – if successful – will result in more than an incremental gain. It is not afraid of going for the “home run.” Donations to many organizations are often used to expand on existing approaches rather than fostering breakthroughs.
GlobalCures enables patients to find their best treatment options today, and thus its work is of immediate value to patients.
GlobalCures views patients and scientists from all over the world as partners in discovery. Unlike much highly specialized research that often happens in discreet silos that may or may not connect with the larger body of research, GlobalCures takes an integrated approach. Its ideas reflect the collective wisdom of the crowds and will result in holistic patient-centered care. Donors who invest in GlobalCures can rest assured that their funds are being to develop effective therapies for patients – not spent on isolated research goals or preconceived ideas that benefit only one approach.
GlobalCures is run by a visionary team, well-connected in scientific and clinical circles.
In summary, GlobalCures is a patient-centered organization whose time has come. It presents a compelling vision with strong leadership that will make a difference to patients today and for the future. The untapped opportunities it explores are novel, affordable and immediately implementable. It will be sustained by the 21st century patient: educated, engaged and empowered. Help yourselves and your children by donating to its cause!
Have you considered requesting some established cancer charities to support GlobalCures?
Yes, GlobalCures intends to approach a number of charities. The pitch will be simple. There are unexplored therapies/drugs that might benefit cancer patients now but they need rigorous validation. That is GlobalCures' major mission: to find these, evaluate them, perhaps combine them and then subject them to careful clinical studies. To date, clinical trials have been supported primarily by the private sector, whereas both the government and cancer charities have largely supported laboratory research. GlobalCures aims to change this paradigm: it is time to support investigator initiated clinical trials that have a sound scientific basis but which industry may not find commercially attractive. Thus, GlobalCures will encourage cancer charities to either support such efforts directly or provide funding to GlobalCures.